Nick Leschly: The Promise of Gene Therapy

In this discussion at Goldman Sachs’ Builders + Innovators Summit, Nick Leschly, the CEO of Bluebird Bio, discusses the progress his company has made in the field of gene therapy to treat and cure disease, from combatting sickle cell disease to progress in the fight against rare forms of cancer, and why understanding our genetics is key to the next generation of groundbreaking medical advancements.

On how gene therapy works: “A way to attack disease is to leverage our understanding of genetics. And so that’s really gene therapy…we manipulate your DNA, but we have to first understand what and why we’re doing it.”

On the success in fighting sickle cell disease: “The idea is that this is a one-time, potentially curative treatment of a terrible, terrible disease that affects hundreds of thousands of people. And so, we’ve had some early luck, and we’ve had some bumps in that, but the first patient we ever treated went from basically going to die very early and [having] a very painful sort of quality of life, to now within six months, not even needing Advil. And so that’s now a couple years out… And this stands on the science of giants.”

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